Status:
ACTIVE_NOT_RECRUITING
Dose-escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects With Retinitis Pigmentosa
Lead Sponsor:
GenSight Biologics
Conditions:
Non-syndromic Retinitis Pigmentosa
Eligibility:
All Genders
18-75 years
Phase:
PHASE1
PHASE2
Brief Summary
The objective of this study is to evaluate the safety and tolerability of escalating doses of a gene therapy called GS030-DP (injected study treatment) administered via a single intravitreal injection...
Detailed Description
Study GS030\_CLIN\_001 is a multicenter, Phase 1/2a, open-label, non-randomized, dose-escalation, safety and tolerability study of GS030-DP in association with GS030-MD in subjects with non-syndromic ...
Eligibility Criteria
Inclusion
- Inclusion criteria:
- Age ≥18 years to ≤75 years at the time of ICF signature.
- Diagnosis of non-syndromic RP defined as:
- Clinical diagnosis of non-syndromic RP based on history, mid-peripheral visual dysfunction, and fundoscopic appearance.
- Diagnosis of non-syndromic RP is confirmed on full-field ERG
- Visual acuity:
- Visual acuity in the dose-escalation cohorts of no better LP.
- Visual acuity in the extension cohort of no better than CF pending review of dose-escalation cohort data by the DSMB.
- Relatively preserved ganglion cell layer volume and retinal nerve fiber layer thickness, as measured with spectral domain optical coherence tomography (SD-OCT).
- Interpupillary distance of ≥51 mm and ≤72 mm.
- Refractive error of the study eye between -6 diopters and +6 diopters.
- Exclusion criteria
- Prior receipt of any gene therapy.
- Subjects who have undergone significant ocular surgery (per investigator determination) within 3 months prior to Visit 1.
- Presence of narrow iridocorneal angles contraindicating pupillary dilation.
- Presence of disorders of the ocular media which interfere with visual acuity and other ocular assessments, including SD-OCT, during the study period.
- Presence of any systemic or ocular diseases, or pathologies, other than non-syndromic RP, or their associated therapies, that can cause or have the potential to cause vision loss.
- Prior vitrectomy or vitreomacular surgery.
- Presence of vitreo-macular adhesion or traction, epiretinal membrane, macular pucker and macular hole, evident by ophthalmoscopy and/or by SD-OCT examinations and assessed by the investigator to significantly affect central vision.
- Current evidence of retinal detachment assessed by the investigator to significantly affect central vision.
- Active ocular inflammation or recurrent history of idiopathic or autoimmune associated uveitis.
- Presence of an Active Implantable Medical Device.
- Subjects who have undergone thermal laser procedure to the retina within 3 months of trial entry, or any prior thermal laser procedure to the macular region.
Exclusion
Key Trial Info
Start Date :
September 26 2018
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 26 2027
Estimated Enrollment :
10 Patients enrolled
Trial Details
Trial ID
NCT03326336
Start Date
September 26 2018
End Date
October 26 2027
Last Update
August 26 2025
Active Locations (3)
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1
UPMC Eye Center
Pittsburgh, Pennsylvania, United States, 15213
2
Centre Hospitalier National d'Ophtalmologie (CHNP) des Quinze-Vingts
Paris, France, 75012
3
Moorfields Eye Hospital NHS Foundation Trust, 162 City Road
London, United Kingdom